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ZIP Study of ATB200/AT2221 in pediatric Pompe disease
jRCT2031210014Recruiting
- Sponsor
- Amicus Therapeutics
- Start
- May 2021
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes, Small Molecules
01
Overview
Open-label, single-arm trial of cipaglucosidase alfa and miglustat in Pompe disease, sponsored by Amicus Therapeutics. Primary endpoint: Safety and tolerability. Started May 2021; status: recruiting.
02
Drugs
cipaglucosidase alfamiglustat
03
Conditions
Pompe disease
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